(19)
(11) EP 4 269 587 A3

(12) EUROPEAN PATENT APPLICATION

(88) Date of publication A3:
10.07.2024 Bulletin 2024/28

(43) Date of publication A2:
01.11.2023 Bulletin 2023/44

(21) Application number: 23185838.2

(22) Date of filing: 19.09.2017
(51) International Patent Classification (IPC): 
C12N 15/113(2010.01)
(52) Cooperative Patent Classification (CPC):
C12N 15/113; C12N 2310/113; C12N 2310/141; A61P 25/14; A61P 21/00; A61K 31/7115
(84) Designated Contracting States:
AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR

(30) Priority: 19.09.2016 ES 201631216

(62) Application number of the earlier application in accordance with Art. 76 EPC:
17776976.7 / 3516059

(71) Applicants:
  • Universitat de València
    46010 Valencia (ES)
  • AUM LifeTech, Inc.
    Philadelphia, PA 19104 (US)

(72) Inventors:
  • ARTERO ALLEPUZ, Rubén D.
    46100 Burjassot, Valencia (ES)
  • LLAMUSÍ TROISI, María Beatriz
    46100 Burjassot, Valencia (ES)
  • CERRO HERREROS, Estefanía
    46100 Burjassot, Valencia (ES)
  • FERNÁNDEZ COSTA, Juan M.
    46100 Burjassot, Valencia (ES)
  • AISHWARYA, Veenu
    Philadelphia, 19104 (US)
  • MØLLER, Thorleif
    5792 Arslev (DK)

(74) Representative: Hoffmann Eitle 
Hoffmann Eitle S.L.U. Paseo de la Castellana 140, 3a planta Edificio LIMA
28046 Madrid
28046 Madrid (ES)

   


(54) MODULATION OF MICRORNAS AGAINST MYOTONIC DYSTROPHY TYPE 1 AND ANTAGONISTS OF MICRORNAS THEREFOR


(57) Modulation of microRNAs against myotonic dystrophy type 1 and antagonists of microRNAs therefor. The invention provides the use of inhibitors of microRNAs repressors of MBNL1 and/or MBNL2 genes for the manufacture of a medicinal product for the treatment of myotonic dystrophy 1. Inhibiting these microRNAs allows to increase the endogenous levels of the corresponding proteins MBNL1 and/or MBNL2, thereby alleviating symptoms of the disease, especially when inhibiting repressors that are expressed in the main affected organs: skeletal muscle, heart or organs of the central nervous system. The inhibition of the microRNAs miR-23b-3p and miR-218-5p is preferred. It also provides oligoribonucleotide or oligoribonucleotide analogue antagonists suitable therefor, preferably antagomiRs directed against the microRNAs mentioned with chemical modifications that enhance their interaction with the target, their stability in vivo and their ability to penetrate into the cells and distribute throughout tissues and organs.





Search report






























Search report